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In Depth Tutorials and Information
for adults living with OI a priority. It influenced among
other things the requirements to become a Linked
Clinical Research Center, and the 2010 and 2012 Science
Meetings.
and resources to devise a feasible and relevant research
agenda and reaches out not just to their patient popu-
lation but to scientists and government representa-
tives of the National Institutes of Health, Food & Drug
Administration and the Centers for Disease Control, to
name a few. These key stakeholders become dedicated
partners in the mission of the Alpha-1 Foundation.
But, the advances in treatments for people living with
the complications of Alpha-1 are only possible because
patients saw and continue to see the value in participat-
ing in research programs.
Large or small, voluntary health agencies adhere
to the basic principles of not-for-management while
utilizing to the best of their ability the most valuable
resource of all…the volunteer. The OI Foundation and
its colleagues in the voluntary health arena value the
volunteer and all that they do to move the mission
forward.
A MODEL THAT WORKS
The work of the OI Foundation, as described, uses a
model of patient advocacy and support that is success-
ful both for large and small not-for-profit organizations
regardless of the staffing structure. Large organizations
with chapters that service specific geographic areas
like the March of Dimes, American Heart Association
or American Cancer Society, partner with medical pro-
fessionals at the local level and utilize their volunteer
networks of professionals to carry out the mission of
the organization as determined at the national level.
These organizations do a very good job of mobilizing
communities through education to make the goals and
objectives of the national organization relevant in their
communities. Small organizations, typically with oper-
ating budgets under $5 million, like the OI Foundation,
carry out their mission in a similar way but depend
much more on their volunteers to serve at a national
level. A good example of a smaller organization that
carries out their mission effectively is the National
Marfan Foundation. Their goal of saving lives and
improving the quality of life for individuals and fami-
lies affected by Marfan syndrome and related disorders
is accomplished by funding research, providing accu-
rate and up-to-date information for patients, families,
health care professionals and the general public and by
providing supporting services to those constituencies.
They have an effective volunteer structure that includes
a national board of directors, professional advisory
board and a scientific advisory board. While they have
chapters across the country, the bulk of the advocacy
work is done at the national level.
A very specific example of how a voluntary health
organization and the patient can partner to make a dif-
ference comes from the Alpha-1 Foundation. Alpha-1
Antitrypsin Deficiency is a genetic condition - it is
passed from parents to their children through their
genes. Alpha-1 may result in serious lung disease in
adults and/or liver disease at any age. In response
to the crisis of not having a treatment for Alpha-1, the
Alpha-1 Foundation mobilized the patient community
as well as the medical community to develop a free
and confidential opportunity for testing for Alpha-1,
established the Alpha-1 Research Registry to encour-
age research, developed new treatments for Alpha-1
and carried out clinical trials to find new treatments
for people living with the complications of Alpha-1.
The Alpha-1 Foundation has spent considerable time
BUT WAIT, THERE'S MORE
In addition to all of the programs and projects
already mentioned, the OI Foundation has sought to
encourage innovation. Its Linked Clinical Research
Center project connects five major OI programs to each
other and to a sophisticated data management system.
The sites conduct joint studies. By linking data, insights
into the natural history of OI will become more evi-
dent more quickly. Analysis of the initial data has led to
updating height and weight charts for children with OI.
The new charts will give parents and their child's doc-
tors a new tool to compare growth to children without
OI plus children with a similar form of OI.
The OI Foundation also sponsors, in partnership
with Kennedy Krieger Institute, an OI Registry. This
program collects contact information and health his-
tory information on children and adults who have OI.
This information is used to help identify topics that
require in-depth study. The contact information is used
to make members aware of opportunities to participate
in research studies they are potentially eligible for. The
Registry is used by researchers as they develop proto-
cols for their research studies. Having a strong patient
advocacy organization enables these types of data col-
lection activities to occur quickly and over an extended
period of time. Having a Registry of people who are
interested in participating in research studies is one
important way to encourage scientists to consider doing
a study on an OI relevant topic.
The Foundation works with volunteers to encour-
age research on critical topics. The Foundation's Adult
Health Initiative came to be because of discussions at
the 2010 OIF Scientific Meeting on the musculoskeletal
issues of OI. A team of medical professionals and adults
